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FDA Grants Accelerated Approval to Lifileucel, First Cellular Therapy for Advanced Melanoma


Lifileucel was granted approval for the treatment of adults with unresectable or metastatic melanoma that has been previously treated with other therapies, marking the first approval of a tumor-derived cellular therapy in a solid tumor cancer.

The FDA today granted accelerated approval to lifileucel (Amtagvi; Iovance Biotherapeutics) for the treatment of adults with unresectable or metastatic melanoma that has been previously treated with other therapies.1

Lifileucel, a tumor-infiltrating lymphocyte therapy, is the first and only cellular therapy to be approved for metastatic melanoma and the only individualized T-cell therapy approved for a solid tumor cancer.2 In an investor call following the approval, Iovance interim CEO Frederick Vogt said the list price for lifileucel would be $515,000 in the US.3

FDA Approved | Image credit: Olivier Le Moal - stock.adobe.com

FDA Approved | Image credit: Olivier Le Moal - stock.adobe.com

“Unresectable or metastatic melanoma is an aggressive form of cancer that can be fatal,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research (CBER), said in a statement.1 “The approval of [lifileucel] represents the culmination of scientific and clinical research efforts leading to a novel T-cell immunotherapy for patients with limited treatment options.”

The accelerated approval is supported by data from the phase 2 C-144-01 trial (NCT02360579), in which 73 patients treated with the recommended dose of lifileucel showed an objective response rate of 31.5% (95% CI, 21.1%-43.4%). This included a complete response rate of 4.1% (n = 3) and a partial response rate of 27.4% (n = 20).

Among those who responded to treatment, 56.5%, 47.8%, and 43.5% maintained responses without tumor progression at 6, 9, and 12 months, respectively. The full trial results were published in The Journal for ImmunoTherapy of Cancer in 2022.1,4

Lifileucel is a tumor-derived autologous T-cell therapy, and this approval marks the first for a tumor-derived T-cell immunotherapy. Tissue from a patient's tumor is surgically removed prior to treatment, T cells are separated from the tissue, and then they are manufactured and reinfused as a single dose.

“Melanoma is a life-threatening cancer that can cause devastating impacts to affected individuals, with a significant risk of metastasizing and spreading to other areas in the body,” said Nicole Verdun, MD, director of the Office of Therapeutic Products in CBER.1 “Today’s approval reflects the FDA’s dedication and commitment to the development of innovative, safe, and effective treatment options for cancer patients.”

Chills, fever, fatigue, tachycardia, diarrhea, febrile neutropenia, edema, rash, hypotension, hair loss, infection, hypoxia, and feeling short of breath were the most common adverse events associated with lifileucel, and the label includes a boxed warning for treatment-related mortality, prolonged severe cytopenia, severe infections, and worsened cardiopulmonary and renal function.


1. FDA approves first cellular therapy to treat patients with unresectable or metastatic melanoma. News release. FDA. February 16, 2024. Accessed February 16, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-first-cellular-therapy-treat-patients-unresectable-or-metastatic-melanoma

2. Iovance’s Amtagvi (lifileucel) receives U.S. FDA accelerated approval for advanced melanoma. News release. Yahoo Finance. February 16, 2024. Accessed February 16, 2024. https://finance.yahoo.com/news/iovance-amtagvi-lifileucel-receives-u-201900117.html

3. Jain P. US FDA grants accelerated approval for Iovance's skin cancer cell therapy. Reuters. February 16, 2024. Accessed February 19, 2024. https://www.reuters.com/business/healthcare-pharmaceuticals/us-fda-grants-accelerated-approval-iovances-skin-cancer-cell-therapy-2024-02-16/

4. Chesney J, Lewis KD, Kluger H, Hamid O, et al. Efficacy and safety of lifileucel, a one-time autologous tumor-infiltrating lymphocyte (TIL) cell therapy, in patients with advanced melanoma after progression on immune checkpoint inhibitors and targeted therapies: pooled analysis of consecutive cohorts of the C-144-01 study. J Immunother Cancer. 2022;10(12):e005755. doi:10.1136/jitc-2022-005755

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